Pain Management in Children and Young Adults With Sickle Cell Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01954927|
Recruitment Status : Completed
First Posted : October 7, 2013
Last Update Posted : July 6, 2018
This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory care setting.
Sickle cell pain is different for every patient. Some patients get complete relief from routine pain medicines, and others need more time or more doses of pain medicines before the pain goes away completely. It is known that humans have many types of pain, including something called neuropathic pain. Neuropathic pain in other conditions (such as diabetes) has been treated successfully with a medicine called gabapentin. The investigators in this study suspect that some sickle cell pain is a combination of pain types. They would like to see if adding gabapentin to the usual pain medicines makes pain go away faster or more completely.
- To assess the analgesic efficacy of gabapentin vs. placebo for pain during vaso-occlusive crisis (VOC) in participants with sickle cell disease (SCD). A response to study drug will be defined by a decrease in pain score of ≥ 33% between presentation to the acute care setting and assessment at 3 hours post administration of study drug.
- To compare the total morphine equivalent dose (mg/kg) used to control pain during VOC between presentation to the acute care setting and assessment at 3 hours post administration of study drug in the gabapentin vs. placebo groups.
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Gabapentin Drug: Placebo||Phase 2|
Upon participant enrollment, study staff will randomize the participant to one of 2 possible treatment arms: a single dose of gabapentin or a single dose of placebo. Morphine or other opioid and non-steroidal anti-inflammatory drugs will be available to both groups as needed for pain and will be administered according to the current standard of care for pain in VOC from the Department of Hematology at St. Jude Children's Research Hospital (SJCRH). Randomization will be performed in the SJCRH pharmacy by a pharmacist. The randomization will be stratified by three age categories (1-3 years of age, 4-6 years, and 7 years or older) for which distinct pain assessment tools are applied and for 2 pain score categories at assessment at presentation (4-6 and 7-10, respectively). A block randomization with block sizes varying randomly between 4 and 6 will be used in each stratum.
Pain scores will be obtained at presentation to the acute care setting and 3 hours (± 15 minutes) post administration of study drug. Participants who were discharged will be contacted by study staff between 24 and 72 hours following administration of study drug to see if there have been any side effects. Patients who were admitted after administration of the study drug will be monitored through hospital record to determine if any unexpected events occurred. After this follow up, participation in the study is complete.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||90 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Pain Management of Vaso-Occlusive Crisis in Children and Young Adults With Sickle Cell Disease|
|Actual Study Start Date :||October 7, 2013|
|Actual Primary Completion Date :||January 3, 2018|
|Actual Study Completion Date :||January 3, 2018|
Active Comparator: Gabapentin
Patients will be randomized to receive one dose of gabapentin.
Gabapentin is supplied as an oral suspension. Patients randomized to the gabapentin arm will receive a single dose of gabapentin as soon after enrollment as feasible. The gabapentin dose will be given orally and will be approximately 15 mg/kg with a maximum dose of 900mg.
Other Name: Neurontin(R)
Placebo Comparator: Placebo
Patients will be randomized to receive one dose of placebo.
Placebo will be prepared by the SJCRH pharmacy and will be similar in appearance, quantity and taste to the gabapentin drug. Patients randomized to the placebo arm will receive a single dose of placebo as soon after enrollment as feasible. The placebo dose will be given orally and will be approximately 15 mg/kg with a maximum dose of 900mg.
- Number and proportion of participants with successful pain interventions by arm [ Time Frame: Baseline and 3 hours post administration of study drug ]Pain scales used are the numerical rating system, the Faces Pain Scale, and the FLACC pain scale (for patients 7 years or older, ages 4-6 years, or less than 4 years, respectively). For each patient, if the reduction of the pain scores (0=no pain and 10=worst possible pain) between presentation to the acute care setting and 3 hours post administration of study drug is 33% or greater, then this patient will be defined as having a successful intervention. The proportions of successful interventions in the gabapentin and placebo groups will be estimated and compared using Z-test.
- Total dose (mg) of opioid analgesic administered [ Time Frame: All doses given between baseline and 3 hours post administration of study drug ]Summary statistics of the total morphine dose or morphine equivalent (mg/kg) used to control pain during VOC between presentation to the acute care setting and 3 hours post administration of study drug, in the gabapentin and placebo groups will be provided. Test of normality such as Shapiro-Wilk test will be applied to the total morphine dose or morphine equivalent (mg/kg) to examine their deviation from the normal distribution. A two-sample t-test or Wilcoxon rank sum test will be used to compare the total morphine dose or morphine equivalent (mg/kg) for the gabapentin vs. placebo groups depending on whether the normality assumption of the data holds.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01954927
|United States, Tennessee|
|St. Jude Children's Research Hospital|
|Memphis, Tennessee, United States, 38105|
|Principal Investigator:||Doralina Anghelescu, MD||St. Jude Children's Research Hospital|